New Therapeutic Approach in an Infant With Systemic Myofibromatosis and Intestinal Hemorrhage.

Christina Salvador, Andreas Entenmann, Roman Crazzolara, Gabriele Kropshofer

Journal: Journal of pediatric hematology/oncology 2022;44(3):109-112

PMID: 34486566

Abstract

We report the case of an infant with multicentric myofibromatosis affecting the gastric and intestinal mucosa, leading to continuous intestinal hemorrhage and iron deficiency. Conventional vinblastine and methotrexate combination treatment was administered for 4 months, but persistent intestinal blood loss required repeated blood transfusions. Because of insufficient tumor response to treatment, we opted for the experimental combination of rapamycin and dasatinib. Six weeks after the start of this therapy, hemoglobin levels stabilized without transfusions, and no fecal blood loss was detected. In addition, a follow-up magnetic resonance imaging excluded tumor progression. We here show the effectiveness of an experimental therapy with rapamycin and dasatinib in a child with multicentric myofibromatosis after the failure of conventional therapy with vinblastine and methotrexate.

Address: Division of Hematology and Oncology, Department of Pediatrics I.; Division of Gastroenterology and Hepatology, Department of Pediatrics I, University Innsbruck, Innsbruck, Austria.

DOI: 10.1097/MPH.0000000000002324

Link outs

Subscription / membership required

Full Text Sources:

MeSH Terms: Child, Dasatinib, Humans, Infant, Methotrexate, Myofibromatosis, Sirolimus, Vinblastine